CA3132111A1 - Antisense nucleic acids for skipping of exon 55 of human dystrophin gene - Google Patents
Antisense nucleic acids for skipping of exon 55 of human dystrophin gene Download PDFInfo
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- CA3132111A1 CA3132111A1 CA3132111A CA3132111A CA3132111A1 CA 3132111 A1 CA3132111 A1 CA 3132111A1 CA 3132111 A CA3132111 A CA 3132111A CA 3132111 A CA3132111 A CA 3132111A CA 3132111 A1 CA3132111 A1 CA 3132111A1
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- Prior art keywords
- exon
- seq
- skipping
- oligomer
- nucleic acid
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- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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Landscapes
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| CA2861247A CA2861247C (en) | 2011-12-28 | 2012-12-27 | Antisense nucleic acids |
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| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| ES2639852T3 (es) | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| CN105779453A (zh) | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| RS58079B1 (sr) | 2009-11-12 | 2019-02-28 | Univ Western Australia | Antisens molekuli i postupci za tretman patologija |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| CN108486116A (zh) * | 2011-12-28 | 2018-09-04 | 日本新药株式会社 | 反义核酸 |
| JP2015509922A (ja) | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド |
| MX366485B (es) | 2013-03-14 | 2019-07-10 | Sarepta Therapeutics Inc | Composiciones para el salto de exón para tratamiento de distrofia muscular. |
| CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
| EP3118311B1 (en) * | 2014-03-12 | 2018-12-26 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
| EP3146970B1 (en) * | 2014-05-19 | 2022-11-02 | KNC Laboratories Co., Ltd. | Nucleic acid drug for inducing skipping of variant exon of cd44 gene and increasing expression of normal type cd44 mrna |
| MY194170A (en) * | 2014-06-17 | 2022-11-16 | Nippon Shinyaku Co Ltd | Antisense nucleic acids |
| WO2016060135A1 (ja) * | 2014-10-14 | 2016-04-21 | 味の素株式会社 | モルフォリノオリゴヌクレオチドの製造方法 |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| LT3351633T (lt) * | 2015-09-15 | 2020-08-10 | Nippon Shinyaku Co., Ltd. | Priešprasmė nukleorūgštis |
| JP2018530560A (ja) | 2015-10-09 | 2018-10-18 | サレプタ セラピューティクス， インコーポレイテッド | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
| WO2017062862A2 (en) | 2015-10-09 | 2017-04-13 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| US10875880B2 (en) * | 2016-05-24 | 2020-12-29 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
| RS65504B1 (sr) * | 2016-05-24 | 2024-06-28 | Sarepta Therapeutics Inc | Procesi za pripremu fosfordiamidat morfolino oligomera |
| US20190262375A1 (en) * | 2016-06-30 | 2019-08-29 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
| WO2018107003A1 (en) * | 2016-12-08 | 2018-06-14 | The Board Of Regents Of The University Of Texas System | Dmd reporter models containing humanized duschene muscular dystrophy mutations |
| HRP20221155T1 (hr) | 2016-12-19 | 2022-12-09 | Sarepta Therapeutics, Inc. | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
| RS65583B1 (sr) | 2016-12-19 | 2024-06-28 | Sarepta Therapeutics Inc | Oligomerni konjugati za preskakanje egzona za mišićnu distrofiju |
| JP7118063B2 (ja) | 2016-12-19 | 2022-08-15 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーコンジュゲート |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| EP3687577A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| US10765760B2 (en) | 2018-05-29 | 2020-09-08 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| US20210308273A1 (en) | 2018-08-02 | 2021-10-07 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| WO2020123574A1 (en) * | 2018-12-13 | 2020-06-18 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| CA3124779A1 (en) * | 2018-12-25 | 2020-07-02 | Nippon Shinyaku Co., Ltd. | Method for inducing muscular cells using cells in spot urine |
| US20230045557A1 (en) | 2019-12-19 | 2023-02-09 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid enabling exon skipping |
| TW202138560A (zh) | 2019-12-26 | 2021-10-16 | 日商日本新藥股份有限公司 | 誘導外顯子５０的跳讀的反義核酸 |
| BR112023021849A2 (pt) | 2021-04-30 | 2023-12-19 | Sarepta Therapeutics Inc | Métodos de tratamento para distrofia muscular |
| US20240287511A1 (en) | 2021-05-13 | 2024-08-29 | National University Corporation Chiba University | Antisense Oligomer |
| KR20240024176A (ko) | 2021-06-23 | 2024-02-23 | 니뽄 신야쿠 가부시키가이샤 | 안티센스 올리고머의 조합 |
| US20240285547A1 (en) | 2021-07-08 | 2024-08-29 | Nippon Shinyaku Co., Ltd. | Nephrotoxicity reducing agent |
| WO2023282346A1 (ja) | 2021-07-08 | 2023-01-12 | 日本新薬株式会社 | 析出抑制剤 |
| CN118475355A (zh) | 2021-07-08 | 2024-08-09 | 日本新药株式会社 | 肾毒性减轻剂 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| WO2023283613A1 (en) * | 2021-07-09 | 2023-01-12 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023026994A1 (ja) | 2021-08-21 | 2023-03-02 | 武田薬品工業株式会社 | ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート |
| AU2022424485A1 (en) | 2021-12-27 | 2024-07-11 | Nippon Shinyaku Co., Ltd. | Method for producing oligonucleic acid compound |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| WO2023171820A1 (ja) * | 2022-03-11 | 2023-09-14 | 日本新薬株式会社 | キャリアペプチドが連結された核酸 |
| WO2023178230A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
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| EP0962463B1 (en) | 1989-12-20 | 2002-07-10 | Antivirals Inc. | Uncharged morpholino-based polymers having phosphorus-containing chiral intersubunit linkages |
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